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Study With SAR302503 in Patients With Polycythemia Vera or Essential Thrombocythemia

NCT01420783 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 81

Last updated 2025-03-05

No results posted yet for this study

Summary

Primary Objective:

* Dose Ranging Phase: To evaluate the efficacy of daily oral doses of 100, 200, and 400 mg SAR302503 in patients with PV and ET who are resistant or intolerant to hydroxyurea (per European LeukemiaNet criteria) for :

* Inducing absence of phlebotomy and a hematocrit below 45% for a minimum of 3 months in patients with polycythemia vera, and
* Reduction of platelet count to ≤400 x 10x9/L for a minimum of 3 months in patients with essential thrombocythemia.
* PV Dose Expansion Phase and ET Dose Ranging Phase (only 600 mg dose group): To evaluate the efficacy of daily oral SAR302503 in patients with PV and ET who are resistant or intolerant to hydroxyurea (per European LeukemiaNet criteria) for:

* Inducing absence of phlebotomy eligibility beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit in patients with PV, and
* Reduction of platelet count to ≤400 x 10x9/L beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit in patients with ET.

Secondary Objectives:

* To evaluate the safety of SAR302503.
* To evaluate the efficacy of SAR302503 in patients with PV who are resistant or intolerant to hydroxyurea for inducing absence of phlebotomy eligibility.
* To evaluate the efficacy of SAR302503 in patients with ET who are resistant or intolerant to hydroxyurea for reduction of platelet counts.
* To evaluate the efficacy of SAR302503 in inducing complete and partial responses beginning at Day 1 of Cycle 6 visit through Cycle 8.
* To evaluate splenic response as measured by spleen volume using MRI or CT.
* To evaluate the pharmacokinetics of SAR302503 after single and repeat doses.
* To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele burden in patients with JAK2V617F mutation, and STAT3 phosphorylation inhibition.
* To measure improvement in baseline myeloproliferative neoplasm (MPN)-associated symptoms, as well as overall impact on quality of life.
* To measure generic health-related quality of life and utility value using the EuroQol Group (EQ-5DTM) questionnaire.

Conditions

  • Hematopoietic Neoplasm

Interventions

DRUG

SAR302503

Pharmaceutical form:capsule Route of administration: oral

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-10-31
Primary Completion
2012-11-30
Completion
2014-05-31

Countries

  • United States
  • Australia
  • Canada
  • France
  • Germany
  • Italy
  • South Korea
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01420783 on ClinicalTrials.gov